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Health, Wellness, Sex and Body Discuss Gene editing saves girl dying from leukaemia in world first at the General Discussion; For the first time ever, a person’s life has been saved by gene editing. One-year-old Layla was dying from leukaemia ...

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Old 11-08-2015, 01:30 AM
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Post Gene editing saves girl dying from leukaemia in world first

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For the first time ever, a person’s life has been saved by gene editing.

One-year-old Layla was dying from leukaemia after all conventional treatments failed. “We didn’t want to give up on our daughter, though, so we asked the doctors to try anything,” her mother Lisa said in a statement released by Great Ormond Street Hospital in London, where Layla (pictured above) was treated.

And they did. Layla’s doctors got permission to use an experimental form of gene therapy using genetically engineered immune cells from a donor. Within a month these cells had killed off all the cancerous cells in her bone marrow.

It is too soon to say she is cured, the team stressed at a press conference in London on 5 November. That will only become clear after a year or two. So far, though, she is doing well and there is no sign of the cancer returning. Other patients are already receiving the same treatment.
https://www.newscientist.com/article...n-world-first/

Sounds promising.
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Old 11-08-2015, 03:40 AM
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Default Re: Gene editing saves girl dying from leukaemia in world first

i remember when leukemia was a death sentence. this is good news for parents
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Old 11-08-2015, 07:28 AM
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Default Re: Gene editing saves girl dying from leukaemia in world first

That's fantastic!
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Old 11-09-2015, 03:21 AM
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Default Re: Gene editing saves girl dying from leukaemia in world first

Yeah!
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Old 02-15-2017, 12:21 AM
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Lightbulb Re: Gene editing saves girl dying from leukaemia in world first

CRISPR-Cas9 may be used to remove genes that cause inherited diseases...

Scientists Soften on DNA Editing of Human Eggs, Sperm, Embryos: Report
February 14, 2017 - Although not ready yet, powerful gene editing tools may one day be used on human embryos, eggs and sperm to remove genes that cause inherited diseases, according to a report by U.S. scientists and ethicists released on Tuesday.
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The report from the National Academy of Sciences (NAS) and the National Academy of Medicine said scientific advances make gene editing in human reproductive cells "a realistic possibility that deserves serious consideration.” The statement signals a softening in approach over the use of the technology known as CRISPR-Cas9, which has opened up new frontiers in genetic medicine because of its ability to modify genes quickly and efficiently. In December 2015, scientists and ethicists at an international meeting held at the NAS in Washington said it would be "irresponsible" to use gene editing technology in human embryos for therapeutic purposes, such as to correct genetic diseases, until safety and efficacy issues are resolved.


A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute to Reuters on May 15, 2012. A group of 25 scientists June 2, 2016, proposed an ambitious project to create a synthetic human genome, or genetic blueprint, in an endeavor that is bound to raise concerns over the extent to which human life can or should be engineered.

The latest NAS report now says clinical trials for genome editing of the human germline could be permitted, "but only for serious conditions under stringent oversight." CRISPR-Cas9 works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA. Genome editing is already being planned for use in clinical trials of people to correct diseases caused by a single gene mutation, such as sickle cell disease. But these therapies affect only the patient.

The concern is over the use of the technology in human reproductive cells or early embryos because the changes would be passed along to offspring. Research using the powerful technique is plowing ahead even as researchers from the University of California and the Broad Institute battle for control over the CRISPR patent. Although gene editing of human reproductive cells to correct inherited diseases "must be approached with caution, caution does not mean prohibition," the committee said in a statement.

Scientists Soften on DNA Editing of Human Eggs, Sperm, Embryos: Report
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Old 04-16-2017, 06:18 AM
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Exclamation Re: Gene editing saves girl dying from leukaemia in world first

WHO says Cancer Incidence Increasing Among Children Worldwide...

Cancer Incidence Increases Among Children Worldwide
April 13, 2017 | WASHINGTON — The number of newly diagnosed childhood cancer cases worldwide rose by 13 percent during the past two decades, according to an agency of the World Health Organization.
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In a study published in the journal The Lancet Oncology, researchers with the International Agency for Research on Cancer in Lyon, France, reported the incidence of childhood cancer was 140 per million per year from 2001-2010 among children up to age 14. The incidence was 124 per million cancers annually throughout the 1980’s, according to data from a previous IARC study. Eva Steliarova-Foucher works in the cancer surveillance section of the IARC, which is part of the WHO. She said cancers that strike adults, notably cancers of the breast, colon and prostate, are often caused by genetic mutations that accumulate over time.

In children, she said, the disease is likely due to a genetic predisposition, adding that children tend to get different cancers than adults. “The first most common cancer in children is leukemia, and this was seen in all the regions. And then it is followed by cancers of the central nervous system in mostly high-income countries, and it was lymphoma in the other world, in low-income countries.” The data were collected from 153 cancer registries in 62 countries, departments and territories covering about 10 percent of the world’s children.


A young cancer patient is seen at a pediatric oncology clinic in Miami, Florida, Dec. 8, 2014. The number of childhood cancer cases rose by 13 percent during past two decades, according to an agency of the World Health Organization.

The best records of childhood cancers were from Western countries, including the United States, which kept records on almost 100 percent of sick children. Five percent or less of the data came from Africa and Asia, according to the report. In those low resource settings, Steliarova-Foucher says many cancers may go undiagnosed because of a lack of awareness and the unavailability of diagnostic equipment.

But she stresses that collection of data is important because, “You need to know how many cases there will be in the next years so that you have enough amenities to take care of these children. You need to know how much their treatment will cost also. So, these data provide the first indicator of the burden (of cancer) in this population.”

For the first time, the IARC report also gathered cancer data on adolescents, between the ages of 15 and 19. The incidence there was 185 cancers in one million teens each year, with lymphoma and melanoma at the top of the list. By knowing the incidence of childhood cancer, Steliarova-Foucher says researchers can begin to identify some of the factors that may contribute to childhood cancer, including environmental pollutants and infections, which might be avoided.

Cancer Incidence Increases Among Children Worldwide
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Old 04-16-2017, 09:13 PM
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Default Re: Gene editing saves girl dying from leukaemia in world first

very cool, very interesting
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Old 07-30-2017, 01:30 AM
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Lightbulb Re: Gene editing saves girl dying from leukaemia in world first

Mebbe dey'll be able to filter out genetic disorders?...

Scientists in US Successfully Edit Human Embryo's Genes
July 27, 2017 - Scientists at the Oregon Health and Science University say they have successfully edited genes of human embryos in the first such attempt in the United States.
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Previously, similar experiments have been reported only by scientists in China. Engineering human genes in the embryo stage opens up the possibility of correcting their defective parts that cause inherited diseases. The new trait is passed on to subsequent generations. But the practice is controversial, since many fear it could be used for unethical purposes such as creating "designer babies" with specific enhanced abilities or traits.


A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute. For the first time, U.S. scientists have successfully edited genes of human embryos.

Oregon scientists led by Kazakhstan-born Shoukhrat Mitalipov successfully repeated the experiment on scores of embryos created with sperm donated for scientific purposes by men with inherited disease mutations. The editing was done very close to the moment of fertilization of the egg in order to make sure the changes would be repeated in all subsequent cells of the embryo.

Scientists have been experimenting with gene editing for a long time, but the availability of the technique called CRISPR rapidly advanced the precision, flexibility and efficiency of cutting and replacing parts of the molecule chains that comprise genes. Citing ethical concerns, the U.S. Congress made it illegal to turn genetically-edited embryos into babies. Many other countries do not have such regulations.

https://www.voanews.com/a/us-scienti...s/3961745.html
See also:

Scientists Soften on DNA Editing of Human Eggs, Sperm, Embryos: Report
February 14, 2017 - Although not ready yet, powerful gene editing tools may one day be used on human embryos, eggs and sperm to remove genes that cause inherited diseases, according to a report by U.S. scientists and ethicists released on Tuesday.
Quote:
The report from the National Academy of Sciences (NAS) and the National Academy of Medicine said scientific advances make gene editing in human reproductive cells "a realistic possibility that deserves serious consideration.” The statement signals a softening in approach over the use of the technology known as CRISPR-Cas9, which has opened up new frontiers in genetic medicine because of its ability to modify genes quickly and efficiently. In December 2015, scientists and ethicists at an international meeting held at the NAS in Washington said it would be "irresponsible" to use gene editing technology in human embryos for therapeutic purposes, such as to correct genetic diseases, until safety and efficacy issues are resolved.

The latest NAS report now says clinical trials for genome editing of the human germline could be permitted, "but only for serious conditions under stringent oversight." CRISPR-Cas9 works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA. Genome editing is already being planned for use in clinical trials of people to correct diseases caused by a single gene mutation, such as sickle cell disease. But these therapies affect only the patient.

The concern is over the use of the technology in human reproductive cells or early embryos because the changes would be passed along to offspring. Research using the powerful technique is plowing ahead even as researchers from the University of California and the Broad Institute battle for control over the CRISPR patent. Although gene editing of human reproductive cells to correct inherited diseases "must be approached with caution, caution does not mean prohibition," the committee said in a statement.

https://www.voanews.com/a/scientists...s/3724179.html
Related:

Stanford Uses CRISPR to Correct Sickle Cell, Human Trials Planned
November 08, 2016 — Scientists at Stanford University School of Medicine have used the CRISPR gene editing tool to repair the gene that causes sickle cell disease in stem cells from diseased patients, paving the way for a potential cure for the disease, which affects up to 5 million people globally.
Quote:
"What we've finally shown is that we can do it. It's not just on the chalkboard," said Dr. Matthew Porteus, senior author of the study published in the journal Nature. With the study, and unpublished findings from his lab, Porteus believes his team has amassed enough proof to start planning the first human clinical trial using the powerful CRISPR-Cas9 gene editing system to correct the genetic mutation that causes sickle cell disease. "We think we have a complete data set to present to the FDA (Food and Drug Administration) to say we've done all pre-clinical experiments to show this is ready for a clinical trial," Porteus told Reuters by phone. CRISPR-Cas9 has quickly become the preferred method of gene editing in research labs because of its ease of use compared with older techniques. CRISPR works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA.

Research using the powerful technique is plowing ahead even as researchers from the University of California and the Broad Institute battle for control over the CRISPR patent. Oral arguments in the case are expected on Dec. 6 at the U.S. Patent and Trademark Office in Alexandria, Va. In sickle cell disease, the body makes mutant, sickle-shaped hemoglobin, the protein in red blood cells that carries oxygen to the body's tissues. It is caused by a single mutation in a gene that makes a hemoglobin protein. In a study published last month in Science Translational Medicine, a team from the University of California, Berkeley, and colleagues used the CRISPR gene editing tool to snip out the diseased gene and deliver a new stretch of DNA to correct the mutation in human stem cells. In that study, some 25 percent of blood-forming cells were corrected.

In the Stanford study, Porteus and colleagues took a different approach. They used CRISPR to snip the gene, but they used a harmless virus to introduce the repair mechanism into cells. After a series of tests in healthy cells, the team tested the gene editing system in blood-forming cells from four patients with sickle cell disease. They showed they could correct the mutation in 30 to 50 percent of these diseased cells. Sixteen weeks after they injected the cells into young mice, the team found the cells were still thriving in the bone marrow. Porteus said the findings were very encouraging because prior studies have shown that if you can correct mutations in 10 percent of cells, that should create enough to cure the disease. Stanford is now scaling up its laboratory processes to support human trials.

The process will involve using chemotherapy to wipe out a patient's blood system but not their immune system, as is done in a stem cell transplant. Then, the team would inject the patient's own corrected stem cells, which the researchers hope would engraft into the bone marrow and produce healthy blood cells. Porteus has equity interest in CRISPR Therapeutics of Cambridge, Massachusetts, but he said the sickle cell work has been independent of it. The university has built a cell manufacturing plant for this purpose. "We hope to develop the entire process here at Stanford," he said. Porteus said the team plans to make an initial submission to the FDA in the next few months to map out the clinical trial, and hopes to treat the first patient in 2018.

https://www.voanews.com/a/reu-stanfo...d/3586115.html
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Old 08-03-2017, 08:20 AM
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Lightbulb Re: Gene editing saves girl dying from leukaemia in world first

Cut-and-Paste Gene Editing...

Like a Cut-and-Paste Tool, Gene Editing Transforms Research
August 02, 2017 | WASHINGTON — Gene editing is getting fresh attention thanks to a successful lab experiment with human embryos. But for all the angst over possibly altering reproduction years from now, this technology already is used by scientists every day in fields ranging from agriculture to drug development.
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New gene editing tools let scientists alter the DNA of living cells - from plants, animals, even humans - more precisely than ever before. Think of it as a biological cut-and-paste program. A look at the science.

What is gene editing?

While scientists have long been able to find defective genes, fixing them has been so cumbersome that it's slowed development of genetic therapies. There are several gene editing methods, but a tool called CRISPR-Cas9 has sparked a boom in research as laboratories worldwide adopted it over the past five years because it's faster, cheaper, simple to use with minimal training and allows manipulation of multiple genes at the same time.

How it works

Pieces of RNA are engineered to be a guide that homes in on the targeted stretch of genetic material. The Cas9 is an enzyme that acts like molecular scissors to snip that spot. That allows scientists to delete, repair, or replace a particular gene.

Medical research

The fresh attention comes from research involving human embryos. In laboratory experiments, a team lead by Oregon researchers used CRISPR to successfully repair a heart-damaging gene in human embryos, marking a step toward one day being able to prevent inherited diseases from being passed on to the next generation. But there's wide agreement that more research is needed before ever testing the technique in pregnancy.


Professor Wendy Harwood poses for a photograph in a plant breeding incubator room with barley plants that have undergone gene editing at the John Innes Centre in Norwich, Britain.

The biggest everyday use of CRISPR so far is to engineer animals with human-like disorders for basic research, such as learning how genes cause disease or influence development and what therapies might help. But promising research, in labs and animals so far, also suggests gene editing might lead to treatments for such diseases as sickle cell, cancer, maybe Huntington's _ by altering cells and returning them to the body. Another project aims to one day grow transplantable human organs inside pigs.

The biggest hurdle
See also:

US Scientists Able to Alter Genes of Human Embryos
August 02, 2017 - U.S. scientists have succeeded in altering the genes of a human embryo to correct a disease-causing mutation, making it possible to prevent the defect from being passed on to future generations.
Quote:
The milestone, published this week in the journal Nature, was confirmed last week by Oregon Health and Science University (OHSU), which collaborated with the Salk Institute and Korea's Institute for Basic Science to use a technique known as CRISPR-Cas9 to correct a genetic mutation for a heart condition. Until now, published studies using the technique had been done in China with mixed results.

CRISPR-Cas9 works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA. "We have demonstrated the possibility to correct mutations in a human embryo in a safe way and with a certain degree of efficiency," said Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory and a co-author of the study.


Juan Carlos Izpisua Belmonte, professor at Salk Institute's Gene Expression Laboratory and Jun Wu, Salk staff scientists

To increase the success rate, his team introduced the genome editing components along with sperm from a male with the targeted gene defect during the in vitro fertilization process. They found that the embryo used the available healthy copy of the gene to repair the mutated part. The Salk/OHSU team also found that its gene correction did not cause any detectable mutations in other parts of the genome - a major concern for gene editing.

Still, the technology was not 100 percent successful - it increased the number of repaired embryos from 50 percent, which would have occurred naturally, to 74 percent. The embryos, tested in the laboratory, were allowed to develop for only a few days. "There is still much to be done to establish the safety of the methods, therefore they should not be adopted clinically," Robin Lovell-Badge, a professor at London's Francis Crick Institute who was not involved in the study, said in a statement.

'Utmost Caution'
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Old 10-25-2017, 03:37 PM
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Cool Re: Gene editing saves girl dying from leukaemia in world first

Studies could lead to new treatments for inherited diseases...

'Incredible' editing of life's building blocks
Wed, 25 Oct 2017 - The studies could lead to new treatments for inherited diseases.
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Scientists have demonstrated an "incredibly powerful" ability to manipulate the building blocks of life in two separate studies. One altered the atoms in DNA to rewrite the human genetic code and the instructions for life. The other edited RNA, which is a chemical cousin of DNA and unlocks the information in the genetic code.


The studies - which could eventually treat diseases - have been described as clever, important and exciting. Cystic fibrosis, inherited blindness and other diseases caused by a single typo in the genetic code could ultimately be prevented or treated with such approaches. Both studies were performed at the Broad Institute of MIT and Harvard.

Base editing

The first, published in the journal Nature, developed tools called base editors. DNA is built out of the four bases: adenine (A), cytosine (C), guanine (G) and thymine (T). If a single one of them is in the wrong place, it can cause disease. Base editors alter the atomic structure of one base to convert it into another. Researchers can now manipulate the four bases. And the team used base editing to correct an inherited disease that leads to dangerously high levels of iron in the blood.

Prof David Liu of the Broad Institute said: "We are hard at work trying to translate base editing technology into human therapeutics." However, he admits there are still issues around safety and implementation: "Having a machine that can make the change you want to make is only the start. You still need to do all this other work, but having the machine really helps."

RNA
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